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Waning treatment for immune deficiency is a warning for all ‘one-and-done’ therapies

The waning effectiveness of bone marrow transplants for a rare immune deficiency is a cautionary tale for anyone developing or receiving a therapy marketed as potentially curative.

As a new mother, she didn’t know to look for blue-tinged lips. She could just tell her baby’s color was off. On a chest X-ray, the clean, white-against-dark curves of his ribs were obscured, clouded by fluid. Pneumonia. That tipped Ray Ballard’s physicians off: He had a form of severe combined immunodeficiency — SCID, for short — a genetic mutation that hampered the growth of crucial immune cells, leaving him utterly vulnerable to infection.

The best fix was a transplant of his mother’s bone marrow. “The attitude was that in three to six months, you should be able to go back to normal life,” recalled his mom, Barb Ballard.

That was true — at least sort of. He got two more booster transplants before he hit 10. An antibiotic left him with hearing loss, and a virus with digestive tract damage. His lack of B cells meant he needed regular injections of other people’s antibodies, and his T cell counts were never ideal. But he was healthy enough to go to public school, to move through the hallways high-fiving half the guys, to slowly inhale and take aim during rifle team practice.

“His T cells had to be working well enough that he wasn’t coming down

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